| Project title | Zero Childhood Cancer: Children’s Cancer Institute Personalised Medicine Program |
|---|---|
Grant Amount | $1.315 million |
Institution | Children’s Cancer Institute |
Investigator Team | Principal investigators Prof. Michelle Haber and A/Prof. David Ziegler |
Grant Type | Clinical Trial |
Years | 2015 – 2019 |
In Australia, 20 children are diagnosed with cancer every week – that is a classroom of children every single week and a thousand kids a year. The challenge of curing them all is that every one of these children is unique and each of them has a cancer that is unique, meaning standard therapies are not effective for every child.
The journey of the Zero Childhood Cancer Program (ZERO) began in 2015 when Children’s Cancer Institute and Kids Cancer Centre at Sydney Children’s Hospital, Randwick, launched Australia’s first-ever precision medicine program for children with cancer. Currently ZERO is open to all Australian children with rare cancer or high-risk cancer (<30% chance of survival), such as those with high-risk brain cancer, which kills more children than any other disease in Australia.
“The funding from CBCF ensured that each brain tumour patient enrolled had access to a comprehensive molecular profiling and when possible, a preclinical testing suite was also established within the ZERO Program.”
Through the collaborative efforts of dedicated researchers and clinicians, we aim to continually improve the outcomes for children with cancer, to one day provide hope to all families affected by childhood cancer.
Progress
Since its launch, ZERO has provided over 600 children nationwide with access to a free-of-charge precision medicine approach to their condition. Over 70% of these patients have since received a potentially actionable precision medicine recommendation based on the comprehensive molecular profiling as well as preclinical modelling and high-throughput drug testing results where possible.
ZERO has also played an important role in improving drug access for Australian children diagnosed with certain types of cancer. As an example, a new phase I clinical trial for the drug Larotrectinib, which targets a specific genetic mutation which drives tumour growth in soft tissue sarcomas. This mutation was identified in one of our ZERO patients via our unique, comprehensive molecular profiling platform and is a clear demonstration of the power of genomically-guided care.
Of the brain tumour patients who have received treatment changes based on ZERO’s results, examples of remarkable clinical responses include:
- A child with a progressive glioblastoma with an FGFR mutation was treated with the FGFR inhibitor ponatinib, which led to 50% shrinkage of their tumour.
- A young adult patient with a Diffuse Midline Glioma with K27M somatic mutation and BRCA2 somatic and germline mutation treated with a PARP inhibitor plus immunotherapy led to complete resolution of his disease.
- A twelve-year-old dying from a metastatic ganglioglioma driven by a BRAFV600E mutation was treated with combined BRAF and MEK inhibitors which led to a complete response.
For some children enrolled on ZERO, they have had more time and an improved quality of life. For a few of these children, ZERO has meant they have survived their cancer to date when they may otherwise have died.
Extensive planning is now underway to expand and enhance the ZERO Program progressively, to reach not only children and young adults with high-risk cancer, but eventually all children diagnosed with cancer regardless of their risk profile.
Our shared goal is that all Australian children and young people with cancer will be able to benefit from ZERO, regardless of the cancer they have by the end of 2023. When this happens, every child diagnosed with cancer in Australia will have access to precision medicine for the very first time.